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A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

NCT04080050 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 8

Last updated 2023-03-09

No results posted yet for this study

Summary

This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease. This study is intended to follow those subjects for up to 5 years since they received treatment to look for any long-term safety concerns. There is no investigational drug or therapy provided as part of this study.

Conditions

  • Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

DRUG

Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy

No investigational product will be administered in this study. All participants have previously received a one-time intravenous infusion of RGX-501 in a separate clinical trial

Sponsors & Collaborators

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-30
Primary Completion
2024-09-29
Completion
2025-09-29
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Netherlands

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04080050 on ClinicalTrials.gov